Immune-Mediated Necrotizing Myopathy
Conditions
Keywords
HMG-CoA Reductase Auto-Antibody Necrotizing Myopathy
Brief summary
This is a phase 2, pilot, randomized, placebo-controlled trial of Gamunex-C IVIG as mono-therapy for HMGCoA reductase auto-antibody positive (HMGCR) necrotizing myopathy. The trial will test the feasibility and initial efficacy of Gamunex-C IVIG mono-therapy in HMGCR necrotizing myopathy.
Detailed description
This is a phase 2, double-blinded, randomized, placebo-controlled, multi-center trial of Gamunex-C IVIG as mono-therapy for HMGCR necrotizing myopathy. Up to 10 treatment-naïve patients will be enrolled and randomized to receive either Gamunex-C IVIG dosed at 2g/kg or placebo at week 0 and week 4. The primary efficacy outcome is the percentage of patients at week 8 with at least minimal improvement per the 2016 ACR/EULAR myositis clinical response criteria.
Interventions
DRUGGamunex-C
Gamunex-C IVIG will be given at week 0 and week 4
DRUGAlbumin
1% albumin solution dosed at equivalent volume to the corresponding weight-based Gamunex-C IVIG dose will be given at week 0 and week 4
Sponsors
Grifols Biologicals, LLC
University of Washington
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
This is a randomized, double-blinded, placebo-controlled phase 2 trial.
Eligibility
Sex/Gender
ALL
Age
18 Years to 120 Years
Healthy volunteers
No
Inclusion criteria
A subject must meet all of the following inclusion criteria at screening to be eligible for participation in this study: * Anti-HMGCR positive. Patients will be screened by commercially-available ELISA. * Age ≥ 18 years * Demonstrable proximal muscle weakness: score of \<135 on the Proximal Manual Muscle Strength Testing 8-Muscle Group Assessment (MMT-8) (range 0-160). * Serum creatinine kinase (CK) more than 5 times the upper limit of normal * Muscle biopsy will not be required for eligibility in order to minimize the time to enrollment and initiation of treatment. Muscle biopsy will be obtained whenever possible as part of the standard of care. * Subjects must be willing and able to provide written informed consent.
Exclusion criteria
A subject meeting any of the following
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ACR/EULAR 2016 Clinical Response Criteria for Myositis | week 8 | The percentage of patients in each arm with at least a minimal response |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of enrolled patients completing all primary and secondary measures | 2 years | — |
| Creatine kinase | week 8 | Serum creatine kinase level will be measured |
| Number of patients screened to achieve planned enrollment | 2 years | — |
| Number of days needed to enroll planned sample | 2 years | — |
| ACR/EULAR Clinical Response Criteria for Myositis Total Improvement Score | week 8 | The total improvement score based on ACR/EULAR 2016 criteria will be calculated. Range 0-100, with higher scores reflecting greater improvement. |
| HMG-CoA Reductase Auto-antibody titer level | week 8 | Autoantibody titer level will be assessed |
| Manual Muscle Strength Testing (MMT-8) score | week 8 | Manual muscle strength testing in 8 muscle groups will be scored. Range 0-160, with higher scores reflecting increased muscle strength. |
Countries
United States
Outcome results
None listed